EP 1567539 A4 20060726 - ALLELE-SPECIFIC RNA INTERFERENCE

Title (en)

ALLELE-SPECIFIC RNA INTERFERENCE

Title (de)

ALLELSPEZIFISCHE RNA-INTERFERENZ

Title (fr)

INTERFERENCE D'ARN PROPRE A UN ALLELE

Publication

EP 1567539 A4 20060726 (EN)

Application

EP 03783121 A 20031104

Priority

US 0335009 W 20031104
US 42350702 P 20021104
US 48828303 P 20030718

Abstract (en)

[origin: WO2004042027A2] Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene. Because the wild type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression. The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wildtype allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations. The invention also includes small interfering RNAs (siRNAs) and Small hairpin RNAs (shRNAs) that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS).

IPC 1-7

C07H 21/04; A61K 48/00; C12N 15/00; C12N 15/11

IPC 8 full level

C12N 5/02 (2006.01); C12N 15/113 (2010.01); A61K 38/00 (2006.01); A61K 48/00 (2006.01)

CPC (source: EP US)

A61P 21/00 (2017.12 - EP); A61P 25/14 (2017.12 - EP); A61P 25/16 (2017.12 - EP); A61P 25/28 (2017.12 - EP); C12N 15/1137 (2013.01 - EP US); C12Y 115/01001 (2013.01 - EP US); A61K 38/00 (2013.01 - EP US); A61K 48/00 (2013.01 - EP US); C12N 2310/111 (2013.01 - EP US); C12N 2310/14 (2013.01 - EP US); C12N 2310/53 (2013.01 - EP US)

Citation (search report)

[PX] WO 03080807 A2 20031002 - HARVARD COLLEGE [US], et al
[Y] WO 9848009 A2 19981029 - UNIV FLORIDA [US]
[X] BRUMMELKAMP T R ET AL: "STABLE SUPPRESSION OF TUMORIGENICITY BY VIRUS-MEDIATED RNA INTERFERENCE", CANCER CELL, XX, US, vol. 2, no. 3, September 2002 (2002-09-01), pages 243 - 247, XP009006464, ISSN: 1535-6108
[Y] FLUITER KEES ET AL: "Tumor genotype-specific growth inhibition in vivo by antisense oligonucleotides against a polymorphic site of the large subunit of human RNA polymerase II.", CANCER RESEARCH. 1 APR 2002, vol. 62, no. 7, 1 April 2002 (2002-04-01), pages 2024 - 2028, XP002380704, ISSN: 0008-5472
[Y] SUI G ET AL: "A DNA vector-based RNAi technology to suppress gene expression in mammalian cells", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE, WASHINGTON, DC, US, vol. 99, no. 8, 16 April 2002 (2002-04-16), pages 5515 - 5520, XP002311179, ISSN: 0027-8424
[A] CAPLEN NATASHA J ET AL: "Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference.", HUMAN MOLECULAR GENETICS. 15 JAN 2002, vol. 11, no. 2, 15 January 2002 (2002-01-15), pages 175 - 184, XP002380705, ISSN: 0964-6906
[A] VICTOR MARTIN ET AL: "HAT activity is essential for CBP-1-dependent transcription and differentiation in Caenorhabditis elegans.", EMBO REPORTS. JAN 2002, vol. 3, no. 1, January 2002 (2002-01-01), pages 50 - 55, XP002380706, ISSN: 1469-221X
See references of WO 2004042027A2

Designated contracting state (EPC)

AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IT LI LU MC NL PT RO SE SI SK TR

DOCDB simple family (publication)

WO 2004042027 A2 20040521; WO 2004042027 A3 20040715; AT E443764 T1 20091015; AU 2003290586 A1 20040607; AU 2003290586 B2 20090702; CA 2504915 A1 20040521; DE 60329416 D1 20091105; EP 1567539 A2 20050831; EP 1567539 A4 20060726; EP 1567539 B1 20090923; ES 2334125 T3 20100305; JP 2006505288 A 20060216; US 2004192629 A1 20040930

DOCDB simple family (application)

US 0335009 W 20031104; AT 03783121 T 20031104; AU 2003290586 A 20031104; CA 2504915 A 20031104; DE 60329416 T 20031104; EP 03783121 A 20031104; ES 03783121 T 20031104; JP 2005502260 A 20031104; US 70081603 A 20031104